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Gene Therapy in Patients With Transfusion-Dependent β-Thalassemia
N. Engl. J. Med 2018 Apr 19;378(16)1479-1493, AA Thompson, MC Walters, J Kwiatkowski, JEJ Rasko, JA Ribeil, S Hongeng, E Magrin, GJ Schiller, E Payen, M Semeraro, D Moshous, F Lefrere, H Puy, P Bourget, A Magnani, L Caccavelli, JS Diana, F Suarez, F Monpoux, V Brousse, C Poirot, C Brouzes, JF Meritet, C Pondarré, Y Beuzard, S Chrétien, T Lefebvre, DT Teachey, U Anurathapan, PJ Ho, C von Kalle, M Kletzel, E Vichinsky, S Soni, G Veres, O Negre, RW Ross, D Davidson, A Petrusich, L Sandler, M Asmal, O Hermine, M De Montalembert, S Hacein-Bey-Abina, S Blanche, P Leboulch, M Cavazzana